Phase 2 Clinical Trial of a Recombinant Adeno-Associated Viral Vector Expressing α(1)-Antitrypsin: Interim Results

Recombinant adeno-associated virus (rAAV) vectors offer promise for the gene therapy of α(1)-antitrypsin (AAT) deficiency. In our prior trial, an rAAV vector expressing human AAT (rAAV1-CB-hAAT) provided sustained, vector-derived AAT expression for >1 year. In the current phase 2 clinical trial,...

Fuld beskrivelse

Na minha lista:
Bibliografiske detaljer
Main Authors: Flotte, Terence R., Trapnell, Bruce C., Humphries, Margaret, Carey, Brenna, Calcedo, Roberto, Rouhani, Farshid, Campbell-Thompson, Martha, Yachnis, Anthony T., Sandhaus, Robert A., McElvaney, Noel G., Mueller, Christian, Messina, Louis M., Wilson, James M., Brantly, Mark, Knop, David R., Ye, Guo-jie, Chulay, Jeffrey D.
Format: Artigo
Sprog:Inglês
Udgivet: Mary Ann Liebert, Inc. 2011
Fag:
Brief Reports
Online adgang:https://ncbi.nlm.nih.gov/pmc/articles/PMC3205788/
https://ncbi.nlm.nih.gov/pubmed/21609134
https://ncbi.nlm.nih.govhttp://dx.doi.org/10.1089/hum.2011.053
Tags: Tilføj Tag
Ingen Tags, Vær først til at tagge denne postø!

Lignende værker